A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 67
- 试验地点
- 27
- 主要终点
- Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
研究概览
简要总结
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- •Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- •Participants with a screening LCI2.5 result ≥7.
- •Participants who are able to swallow tablets.
排除标准
- •Clinically significant cirrhosis with or without portal hypertension.
- •Colonization with organisms associated with a more rapid decline in pulmonary status.
- •Solid organ or hematological transplantation.
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Placebo
Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
干预措施: Placebo (Drug)
TEZ/IVA
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
干预措施: TEZ/IVA (Drug)
TEZ/IVA
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
干预措施: IVA (Drug)
TEZ/IVA
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
干预措施: Placebo (Drug)
Ivacaftor
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
干预措施: IVA (Drug)
Ivacaftor
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
时间窗: From baseline through Week 8
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
次要结局
- Absolute Change in Sweat Chloride At Week 8(From baseline at Week 8)
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8(From baseline through Week 8)
- Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit(From first dose of study drug up to safety follow-up visit (up to Week 12))